Marisa Wexler, MS,  senior science writer—

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

A Phase 2 clinical trial of oral lenabasum in people with cystic fibrosis (CF) failed to meet its primary goal of reducing pulmonary exacerbations, the investigational therapy’s developer, Corbus Pharmaceuticals, announced. “We are very disappointed that the study did not meet the primary endpoint,” Barbara White, MD, chief medical officer and…

Oral antibiotics are just as effective as intravenous (into-the-vein) antibiotics at killing Pseudomonas aeruginosa bacteria in people with cystic fibrosis (CF), a new study shows. This finding may allow people with CF to spend less time in the hospital to receive treatment, ultimately reducing treatment burden. The study,…

The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) as a treatment for infants as young as four months with cystic fibrosis (CF) caused by certain mutations. The approval makes Kalydeco the first medication in its class to be approved for…

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended extending the licenses for Symkevi and Kalydeco, two cystic fibrosis (CF) treatments marketed by Vertex Pharmaceuticals, to allow these medications to be used in younger children. If approved by…

Adding pancreatic enzymes to “pre-digest” food was found to be an effective way to ensure nutrition in an infant with cystic fibrosis who was being fed through a feeding tube, according to a recent case report. The report, “Crushing pancreatic enzymes with enteral feeds in an extremely…

The European Medicines Agency (EMA) has validated an application that seeks to expand the approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in individuals 12 and older who have at least one copy of the F508del mutation…

Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is safe for use in children with cystic fibrosis (CF) as young as age 6, data from a global Phase 3 clinical trial show. Based on these results, Vertex Pharmaceuticals, which markets Trikafta, will seek an expansion of the medication’s current U.S.

A series of new research projects will seek to better understand how the immune system responds to transplanted organs, including the lungs. The goal is to find ways to increase the viability of organs after transplant, which could improve care for conditions like cystic fibrosis (CF). The projects are…

A new strategy for detecting very small concentrations of specific molecules could allow for better diagnosis and management of a variety of diseases, including cystic fibrosis (CF). The method was described in the journal Nature Communication by a team from the University of Leeds, U.K., in a study titled…